Down syndrome

SE Antonarakis, BG Skotko, MS Rafii… - Nature Reviews …, 2020 - nature.com
Trisomy 21, the presence of a supernumerary chromosome 21, results in a collection of
clinical features commonly known as Down syndrome (DS). DS is among the most …

Opportunities, barriers, and recommendations in Down syndrome research

JA Hendrix, A Amon, L Abbeduto… - … science of rare …, 2021 - journals.sagepub.com
BACKGROUND: Recent advances in medical care have increased life expectancy and
improved the quality of life for people with Down syndrome (DS). These advances are the …

A non-mosaic transchromosomic mouse model of down syndrome carrying the long arm of human chromosome 21

Y Kazuki, FJ Gao, Y Li, AJ Moyer, B Devenney… - Elife, 2020 - elifesciences.org
Animal models of Down syndrome (DS), trisomic for human chromosome 21 (HSA21) genes
or orthologs, provide insights into better understanding and treatment options. The only …

Improving public health requires inclusion of underrepresented populations in research

CY Spong, DW Bianchi - Jama, 2018 - jamanetwork.com
Advances in genomicshaveusheredinpromisingthera-pies tailored to the individual.
Personalized medicine is promoted and has begun to positively influence care. For …

Neurodevelopment in Down syndrome: Concordance in humans and models

JA Klein, TF Haydar - Frontiers in cellular neuroscience, 2022 - frontiersin.org
Great strides have been made over the past 30 years in understanding the
neurodevelopmental changes underlying the intellectual disability (ID) in Down syndrome …

Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: Feasibility, practice effects, test-retest reliability, and …

L Abbeduto, E Berry-Kravis, A Sterling… - Journal of …, 2020 - Springer
Background The evaluation of treatment efficacy for individuals with fragile X syndrome
(FXS) or intellectual disability (ID) more generally has been hampered by the lack of …

A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down …

C Goeldner, PS Kishnani, BG Skotko, JL Casero… - Journal of …, 2022 - Springer
Background There are currently no pharmacological therapies to address the intellectual
disability associated with Down syndrome. Excitatory/inhibitory imbalance has been …

Aging with Down syndrome—where are we now and where are we going?

MJ Alldred, AC Martini, D Patterson, J Hendrix… - Journal of clinical …, 2021 - mdpi.com
Down syndrome (DS) is a form of accelerated aging, and people with DS are highly prone to
aging-related conditions that include vascular and neurological disorders. Due to the …

Prenatal and postnatal pharmacotherapy in Down syndrome: the search to prevent or ameliorate neurodevelopmental and neurodegenerative disorders

R Bartesaghi, S Vicari… - Annual review of …, 2022 - annualreviews.org
Those with Down syndrome (DS)—trisomy for chromosome 21—are routinely impacted by
cognitive dysfunction and behavioral challenges in children and adults and Alzheimer's …

Spoken language outcome measures for treatment studies in Down syndrome: Feasibility, practice effects, test-retest reliability, and construct validity of variables …

AJ Thurman, JO Edgin, SL Sherman, A Sterling… - Journal of …, 2021 - Springer
Background The purpose of this study was to evaluate expressive language sampling (ELS)
as a procedure for generating spoken language outcome measures for treatment research …